A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection

Hemophilia A Clinical Trial

Official title:

A Phase I, Multicentre, Open-label Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection in Children With Severe Hemophilia A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05251090
• Other study ID #: CTR20220279
• Status: Completed
• Phase: Phase 1
• Start date: June 16, 2021
• Est. completion date: May 9, 2022

Study information

• Verified date: August 2023
• Source: Jiangsu Gensciences lnc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: May 9, 2022
• Est. primary completion date: November 15, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 12 Years

Eligibility

Key Inclusion Criteria:

• The activity of the coagulation factor VIII (FVIII:C) < 1%. Less than 6 years old Patients previously treated with FVIII concentrate (s) for a minimum of 50 exposure days (EDs) prior to study entry. 6 years old to 12 years old Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.

• Normal prothrombin time or INR < 1.3.

• Negative lupus anticoagulant.

Key Exclusion Criteria:

• Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins).

• History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration.

• Current FVIII inhibitor-positive or history of FVIII inhibitor-positive.

• Other coagulation disorder(s) in addition to hemophilia A.

• Infusion of any products containing FVIII within 72 h prior to administration.

• Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), BUN > 2×ULN, Cr > 2.0 ULN).

• One or more clinically significant tests for Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody.

• Patients who received any anticoagulant or antiplatelet therapy within one week prior screening or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials.

• Patients having major surgery or receiving blood or bood components transfusion within 4 weeks prior screening or having planned major surgery schedule during the study.

• Patients who previously participated in the other clinical trials within one month prior to administration.

• Any life-threatening disease or condition which, according to the investigator's judgment, could not benefit from the trial participation.

• Patient who is considered by the other investigators not suitable for clinical study.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• ADVATE
50 international units (IU)/kg, a single dose.
• FRSW107
50 IU/kg, a single dose.

Locations

Country	Name	City	State
China	Beijing Children's Hospital,Capital Medical University	Beijing	Beijing
China	Chengdu Women's and Children's Central Hospital	Chengdu	Sichuan
China	Nanfang Hospital of Southern Medical University	Guangzhou	Guangzhou
China	The Affiliated Hospital of Qingdao University	Qingdao	Shandong
China	Shenzhen Children's Hospita	Shenzhen	Guangdong
China	Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology	Wuhan	Hubei

Sponsors (1)

Lead Sponsor	Collaborator
Jiangsu Gensciences lnc.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximum measured concentration of FVIII:C (Cmax)	Measured by aPTT Clotting Assay.	Pre-dose and post dose up to 8 days.
Primary	Time required for the concentration of the drug to reach half of its original value (T1/2)	Measured by aPTT Clotting Assay.	Pre-dose and post dose up to 8 days
Primary	Area Under the Curve to Infinity (AUC)	Measured by aPTT Clotting Assay.	Pre-dose and post dose up to 8 days.
Primary	The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL).	Measured by aPTT Clotting Assay.	Pre-dose and post dose up to 8 days.
Secondary	Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.	Adverse events related to Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0.	Post dose up to 32 days.
Secondary	Development of Inhibitor	Measured by the Nijmegen-Modified Bethesda Assay.	Pre-dose and post dose up to 32 days.